European Medicines Agency Validates Amicus Therapeutics Marketing Authorization Applications for AT-GAA for the Treatment of Pompe Disease
December 03, 2021 at 07:00 am EST
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Amicus Therapeutics, Inc. announced that the European Medicines Agency (EMA) validated the Marketing Authorization Applications (MAA) for AT-GAA, the Company?s investigational two-component therapy for the treatment of Pompe disease. Validation of the application confirms the submission is accepted, and the EMA?s centralized procedure with Committee for Medicinal Products for Human Use (CHMP)?s assessment begins. The MAAs were submitted to the EMA based on the evaluation of the effects of AT-GAA in adults living with Pompe disease and its safety profile, which include data from the Phase 1/2 and Phase 3 PROPEL studies, as well as data from the long-term open-label extension study.
Amicus Therapeutics, Inc. is a biotechnology company, which is focused on discovering, developing, and delivering novel medicines for rare diseases. Its two marketed therapies are Galafold, the first oral monotherapy for people living with Fabry disease who have amenable genetic variants, and Pombiliti + Opfolda, a novel treatment designed to improve uptake of active enzyme into key disease relevant tissues for adults living with late-onset Pompe disease. As an orally administered monotherapy, Galafold is designed to bind to and stabilize an endogenous alpha-galactosidase A (alpha-Gal A) enzyme in those patients with genetic variants identified as amenable in a Good Laboratory Practice (GLP) cell-based amenability assay. Pombiliti + Opfolda consists of a uniquely engineered rhGAA enzyme, cipaglucosidase alfa-atga, with an optimized carbohydrate structure to enhance lysosomal uptake, administered in combination with miglustat that functions as an enzyme stabilizer.
AMICUS THERAPEUTICS, INC. 
